Applications of the Single Base Editing Technology in the Treatment of Hereditary Anemia

VernacularTitle:单碱基编辑技术在治疗遗传性贫血中的应用
Author: Li-Yuan WANG ¹ ; Yu-Yu FU ; Yuan-Bin XIE
Author Information

1. 赣南医科大学基础医学院,江西赣州 341000
Keywords: single base editor(BEs); hereditary anemia; gene mutation
From: Chinese Journal of Biochemistry and Molecular Biology 2024;40(6):770-778
CountryChina
Language:Chinese
Abstract: Single base editors(BEs)are a new class of base editors derived from the CRISPR/Cas sys-tem or transcription activator-like effector(TALE)gene editing technology,which can accurately edit a single base without introducing DNA double-strand-breaks or donor DNA.Compared with the previous genome editing technologies,it has the advantages of less by-products,higher editing efficiency,and lar-ger base editing range,which can be used for the treatment of genetic anemia caused by point mutations.In this paper,the development of single base editing technology and its progress in gene therapy for trea-ting genetic anemia caused by gene mutations are summarized,which can provide a theoretical reference for the clinical trial of gene therapy for clinical anemia.